Final Results from Phase 2 Combination Study of Kalydeco and VX-809 Show Significant Improvements in Lung Function

June 28, 2012

A Phase 2 clinical trial of Kalydeco™ in combination with VX-809 showed significant improvements in lung function in people with the most common CF mutation, according to final results announced today by Vertex Pharmaceuticals Inc.

Both Kalydeco and VX-809, a CF drug in development, are designed to treat the root cause of cystic fibrosis. Vertex developed Kalydeco and VX-809 with significant financial, clinical and scientific support from the Cystic Fibrosis Foundation.

The 56-day study enrolled 109 people, ages 18 and older, with one or two copies of Delta F508. People in the study with two copies of Delta F508 (the most common CF mutation) who received the highest dose of VX-809 combined with Kalydeco showed the greatest improvement in lung function. Vertex released preliminary results from the Phase 2 trial earlier this year.

Based on these final results, Vertex plans to begin a pivotal trial of the combination treatment in people with two copies of Delta F508 in early 2013. Pivotal trials typically aim to gather data that the U.S. Food and Drug Administration (FDA) could use to decide whether to approve a potential drug.

“The improvements seen in lung function are very encouraging, and we are pleased that Vertex plans to move forward quickly with a pivotal trial,” said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. “We still have significant work ahead of us, but the entire CF community can take pride in its role in making this important step possible. We thank the trial volunteers and their families, clinicians and scientists, and our dedicated volunteers and donors across the country.”

Participants with one copy of the Delta F508 mutation also showed improvements in lung function, compared with those who received a placebo — though smaller than the improvements seen in those with two copies of Delta F508. Vertex said it plans to conduct additional studies of Kalydeco and VX-809 in those with one copy of Delta F508.

About 50 percent of people with CF in the United States have two copies of the Delta F508 mutation; an additional 40 percent of people in the United States have one copy.

The FDA approved Kalydeco in January 2012 for people with the G551D mutation of CF ages 6 and older. About 4 percent of people in the United States have the G551D mutation.

People with CF and their families who have questions about the Phase 2 results can contact Vertex Medical Information at 1-877-634-8789.